Hydroxypropyl beta-cyclodextrin compositions and methods
AssigneeMandos LLC
InventorsBernardus Nicolaas Machielse, Allan Darling
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Abstract
This disclosure provides mixtures of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, the mixture optionally including unsubstituted beta-cyclodextrin molecules, for use as a pharmaceutically active ingredient; methods of making such mixtures; methods of qualifying such mixtures for use in a pharmaceutical composition suitable for intrathecal or intracerebroventricular administration; pharmaceutical compositions suitable for intrathecal or intracerebroventricular administration comprising such mixtures; and methods of using the pharmaceutical compositions for treatment of Niemann-Pick disease Type C.
Claims23
A composition comprising a mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein:
- the mixture comprises less than 1% unsubstituted beta-cyclodextrin (“DS-0”) and beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”);
- and, at least 70% of the beta-cyclodextrins have a DS within DSa±1σ, wherein σ is the standard deviation of the DS range, wherein DSa is the average degree of substitution, and wherein the composition comprises no more than 0.01% propylene glycol.
Dependencies are inferred from the claim text.